Single IV Treatment Retrains Immune Cells to Combat Autoimmune Diseases in Early Trial
Researchers have developed a novel, single intravenous (IV) treatment, JY231, that retrains a patient's immune cells to fight severe neurological and muscle autoimmune diseases. This new approach aims to simplify CAR T-cell therapy, which traditionally involves extracting, genetically modifying, and reinfusing a patient's T cells in a complex and time-consuming laboratory process. JY231 utilizes a harmless engineered lentiviral vector to deliver genetic instructions directly into the body, reprogramming the patient's own T cells to target CD19, a protein found on B cells responsible for autoimmune attacks. In an early clinical trial, 16 patients with hard-to-treat conditions such as progressive multiple sclerosis (MS), myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD), generalized myasthenia gravis (gMG), and idiopathic inflammatory myopathies (IIM) received a single infusion. The treatment successfully generated CAR-T cells internally, leading to a rapid, temporary depletion of B cel...